Fibrodysplasia Ossificans ProgressivaNCT04665323
An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.
An observational study of Fibrodysplasia Ossificans Progressiva, run by Ipsen at 1 site in United States.
Trial summary
The FOP burden of illness (BoI) survey aims to assess the impact of the burden of FOP on patients and their families. The study is being conducted online and available for residents in Argentina, Brazil, Canada, France, Germany, Italy, Japan, Mexico, Poland, Russia, South Korea, Spain, Sweden, the US, and the UK. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fibrodysplasia Ossificans Progressiva
- How many people
- 411 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Jan 2021to 30 Apr 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Convenience Sample
Who may be able to join
- Individuals meeting one of the two categories below (A or B) are eligible to participate: A. Any individual with FOP, of any age; B. Any individual who is a family member of a person with FOP (i.e. either a parent / legal guardian or a sibling) and who is aged 18 years and older
- All adult participants and parents / legal guardians of minors should provide informed consent before starting filling out the survey
Who cannot join
- 1. Any individuals who are unable to complete the online survey independently or to get assistance to physically enter the answers
Trial locations and contacts
1 site in 1 country.
United States1 site
Ipsen Central Contact
Cambridge, Massachusetts, 02142
About this study
The FOP burden of illness (BoI) survey aims to assess the impact of the burden of FOP on patients and their families. The study is being conducted online and available for residents in Argentina, Brazil, Canada, France, Germany, Italy, Japan, Mexico, Poland, Russia, South Korea, Spain, Sweden, the US, and the UK.
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Medical subject headings
- Myositis Ossificans
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| People living with FOP | Not described | |
| Parent or legal guardian primary caregivers | Not described | |
| Parent or legal guardian | Not described | |
| Siblings | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quality of life of people living with FOP and their family members, measured using the EuroQol health-related quality of life (QoL) questionnaire (EQ-5D-5L)
Measured: Baseline
Quality of life of people living with FOP and their family members, measured using the Patient-Reported Outcomes Measurement Information System (PROMIS)
Measured: Baseline
Caregiver burden for the parent primary caregiver, measured using the Zarit Burden Interview (ZBI)
Measured: Baseline
Responses to bespoke questionnaire describing the emotional burden on family members
Measured: Baseline
Physical function of the person living with FOP, measured using the FOP Physical Function Questionnaire (FOP-PFQ).
Measured: Baseline
Joint function of the person living with FOP, measured using Patient-Reported Mobility Assessment (PRMA).
Measured: Baseline
Responses to bespoke questionnaire describing the types of healthcare services utilized by the person living with FOP over the last two years
Measured: Baseline
Responses to bespoke questionnaire describing the frequency of utilization of healthcare services by the person living with FOP over the last two years.
Measured: Baseline
Responses to bespoke questionnaire describing the impact of FOP on modifications to the living environment (e.g. home modifications) and travels.
Measured: Baseline
Responses to bespoke questionnaire describing the expenses paid by the family for the care of the person living with FOP
Measured: Baseline
Responses to bespoke questionnaire describing the Impact of FOP on education, employment, and career choices for people living with FOP and their family members
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2020
- Study start18 Jan 2021
- Primary completion30 Apr 2021
- Study completion30 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fibrodysplasia Ossificans ProgressivaStudies of the same condition, open and under way.
- More trials run by IpsenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 411 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04665323
- Sponsor's protocol ID
- CLIN-60120-450
- Status last verified
- May 2021
- First submitted
- 25 Nov 2020
- Last updated
- 21 May 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.
- ClinicalTrials.gov ID
- NCT04665323
- Acronym / sponsor ID
- CLIN-60120-450
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 411 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- 18 Jan 2021 (actual)
- Primary completion
- 30 Apr 2021 (actual)
- Study completion
- 30 Apr 2021 (actual)
- First posted
- 11 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The FOP burden of illness (BoI) survey aims to assess the impact of the burden of FOP on patients and their families. The study is being conducted online and available for residents in Argentina, Brazil, Canada, France, Germany, Italy, Japan, Mexico, Poland, Russia, South Korea, Spain, Sweden, the US, and the UK.
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| People living with FOP | – | – |
| Parent or legal guardian primary caregivers | – | – |
| Parent or legal guardian | – | – |
| Siblings | – | – |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Convenience Sample
Key inclusion criteria (2 listed)
- Individuals meeting one of the two categories below (A or B) are eligible to participate: A. Any individual with FOP, of any age; B. Any individual who is a family member of a person with FOP (i.e. either a parent / legal guardian or a sibling) and who is aged 18 years and older
- All adult participants and parents / legal guardians of minors should provide informed consent before starting filling out the survey
Key exclusion criteria (1 listed)
- 1. Any individuals who are unable to complete the online survey independently or to get assistance to physically enter the answers
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Quality of life of people living with FOP and their family members, measured using the EuroQol health-related quality of life (QoL) questionnaire (EQ-5D-5L)
Time frame: Baseline
Quality of life of people living with FOP and their family members, measured using the Patient-Reported Outcomes Measurement Information System (PROMIS)
Time frame: Baseline
Caregiver burden for the parent primary caregiver, measured using the Zarit Burden Interview (ZBI)
Time frame: Baseline
Responses to bespoke questionnaire describing the emotional burden on family members
Time frame: Baseline
Physical function of the person living with FOP, measured using the FOP Physical Function Questionnaire (FOP-PFQ).
Time frame: Baseline
Joint function of the person living with FOP, measured using Patient-Reported Mobility Assessment (PRMA).
Time frame: Baseline
Responses to bespoke questionnaire describing the types of healthcare services utilized by the person living with FOP over the last two years
Time frame: Baseline
Responses to bespoke questionnaire describing the frequency of utilization of healthcare services by the person living with FOP over the last two years.
Time frame: Baseline
Responses to bespoke questionnaire describing the impact of FOP on modifications to the living environment (e.g. home modifications) and travels.
Time frame: Baseline
Responses to bespoke questionnaire describing the expenses paid by the family for the care of the person living with FOP
Time frame: Baseline
Responses to bespoke questionnaire describing the Impact of FOP on education, employment, and career choices for people living with FOP and their family members
Time frame: Baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ipsen Medical Director study director
Ipsen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fibrodysplasia Ossificans Progressiva, run by Ipsen at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quality of life of people living with FOP and their family members, measured using the EuroQol health-related quality of life (QoL) questionnaire (EQ-5D-5L) (time frame: Baseline). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 May 2021.